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AVROBIO`s vision is to bring personalized gene therapy to the world. We aim to prevent, halt or reverse disease throughout the body with a single dose of gene therapy designed to drive durable expression of therapeutic protein, even in hard-to-reach tissues and organs including brain, muscle and bone. Our ex vivo lentiviral gene therapy pipeline includes clinical programs in Fabry disease, Gaucher disease type 1 and cystinosis, as well as preclinical programs in Hunter syndrome, Gaucher disease type 3 and Pompe disease. AVROBIO is powered by our industry leading plato® gene therapy platform, our foundation designed to deliver gene therapy worldwide. We are headquartered in Cambridge, Mass., with an office in Toronto, Ontario.
Syndax is a clinical stage biopharmaceutical company developing entinostat as a combination therapy in multiple cancer indications with an initial focus on tumors that have shown sensitivity to immunotherapy, including lung cancer, melanoma, ovarian cancer and triple-negative breast cancer (TNBC). Entinostat is an oral, small molecule drug candidate that has direct effects on both cancer cells and immune regulatory cells, potentially enhancing the body`s immune response to tumors. Entinostat is being evaluated as a combination therapeutic in Phase 1b/2 clinical trials with Merck and Co., Inc. for non-small cell lung cancer and melanoma, with Genentech, Inc. for TNBC, and with Merck KGaA, Darmstadt, Germany and Pfizer Inc. in ovarian cancer. Syndax is also developing entinostat as a combination therapeutic in a Phase 3 clinical trial that is being conducted with ECOG-ACRIN for advanced hormone receptor positive breast cancer.
Sangamo Therapeutics, Inc. is focused on translating ground-breaking science into genomic therapies that transform patients` lives using the company`s industry leading platform technologies in genome editing, gene therapy, gene regulation and cell therapy. The Company is conducting Phase 1/2 clinical trials in Hemophilia A and Hemophilia B, and lysosomal storage disorders MPS I and MPS II. Sangamo has an exclusive, global collaboration and license agreement with Pfizer Inc. for gene therapy programs for Hemophilia A, with Bioverativ Inc. for hemoglobinopathies, including beta thalassemia and sickle cell disease, and with Shire International GmbH to develop therapeutics for Huntington`s disease. In addition, it has established strategic partnerships with companies in non-therapeutic applications of its technology, including Sigma-Aldrich Corporation and Dow AgroSciences.
Aptinyx Inc. (formerly Naurex Inc.) is a biopharmaceutical company focused on discovery and development of transformative therapies for challenging disorders of the brain and nervous system. Aptinyx has a proven platform for discovering compounds that enhance synaptic plasticity, or strengthen the network for neural cell communication. Molecules discovered by Aptinyx achieve this through a novel mechanism that modulates NMDA receptors, resulting in drugs that are both highly effective and well tolerated. This mechanism has applicability across a number of brain and nervous system disorders. Aptinyx is rapidly advancing promising drug candidates into clinical development for indications with high unmet medical need.
Curemark, LLC is a clinical-stage biopharmaceutical company focused on developing novel therapies for the treatment of neurological disorders. Curemark`s pipeline includes preclinical and clinical-stage programs for the treatment of Autism, ADHD, addiction, Schizophrenia and Parkinson`s disease.